Outsiders, Sleepers, and Early Stagers
Like last year, some of the products uncovered during the research phase of this year's pipeline report weren't easily shoehorned
into the categories presented, for one reason or another. Here are few category outsiders, sleepers—defined as potential big
sellers people aren't talking about—and early stage drugs that look solid, but haven't gotten far enough in the clinic to
prompt blockbuster whispers.
» Amorcyte's (acquired by NeoStem in 2011) AMR 001 drug candidate, an early-stager, is a bone marrow-derived stem cell product
in development for the emergency treatment of severe heart attack. The company is assessing CD34-positive autologous stem
cells (autologous means the stem cells come from the same patient they're used to treat) that would potentially improve health
outcomes following a severe heart attach. Results from the Phase II trial are expected during the first half of next year.
Martin Sullivan at INC Research says next-gen cell therapies like AMR 001 represent the next generation of better cell therapies,
a promising development
» Novartis's secukinumab is an Isle 17 monoclonal antibody for the treatment of psoriasis. A sleeper because psoriasis is
often in the shadow of rheumatoid arthritis, at least from a biologic drug perspective, secukinumab could wake up investors
with blockbuster sales as soon as 2018, according to several forecasts. In Phase III trials, Novartis had to toss out the
psoriasis area and severity index (PASI) 75 scale, because everyone in the trial passed the 75 percent mark, says Ben Weintraub,
» Even as an early-stager, Oramed Pharmaceuticals' ORMD 0801 is ahead of the big diabetes shops in the race to oral insulin
for diabetes. Oramed is pursuing a Type 2 indication first, for patients who don't want to transition to injected insulin,
but the company has also launched a trial studying ORMD 0801 in Type 1 insulin dependent patients. Raghuram Selvaraju, at
Aegis Capital Corp., says if ORMD 0801 finds its way to market in the next four or five years, "it would blow [MannKind's]
Afrezza away." Liquid (and Afrezza's powedered) insulin must be kept refrigerated; an oral version, particularly without the
need to keep it cold, could be huge in markets with access issues.
» Merck's suvorexant, a hypnotic and outsider, has been filed in the United States and Japan for the treatment of insomnia.
But FDA denied the application last summer in a Complete Response Letter, over worries that the dosage was too large. Merck
plans to resubmit in the first half of 2014; FDA did not require additional trials. The consensus among analysts prior to
the CRL was blockbuster sales, by 2019. Given the relatively short hold up in resubmitting, Merck may still get a sweet dream
or two out of suvorexant.
» GW Pharmaceuticals' sleeper product Sativex is probably the first drug derived from raw cannabis to cross the FDA's desk
at least since marijuana was slapped with a schedule 1 classification in 1970. Schedule 1 drugs are considered to have "no
currently accepted medical use." Sativex is approved in 22 countries, but in the United States, Phase III trials for a cancer
pain indication will conclude next year, with an NDA to follow in early 2015, according to GW Pharma CEO Justin Gover. Credit
Suisse puts sales at roughly half a billion by 2020.
» Synageva Biopharma's sebelipase alfa is an outsider in Phase III for the treatment of Wolman disease, a rare lisosomal storage
disorder in which patients are unable to break down certain lipids. Most infants with the disease don't survive more than
a year, and adults with the disease have problems with swelling of the abdomen, liver enlargement and serious, life-threatening
digestive problems. Sebelipase alfa would become the first approved treatment for Wolman disease, and FDA has granted Orphan
Drug, Fast Track and Breakthrough Therapies status. Given the pricing environment for orphan drugs, Synageva Biopharma could
be rewarded handsomely.
Ben Comer is Pharm Exec's Senior Editor. He can be reached at firstname.lastname@example.org
Forecasting data in Pharm Exec's 2014 Pipeline Report relies in part on Springer's Adis R&D Insight database, Thomson Reuters
Cortellis, Credit Suisse, and Evaluate Pharma data. We very much appreciate the use of these resources.